Patient voice in clinical development: Can patients contribute to the benefit-risk assessment of new medicines?
13-14 March 2013, Surrey, UK
The consensus of the April 2012 CIRS Workshop, The Patient’s Role in the Benefit-Risk Assessment for the Submission and Review of New Medicines, was that patients should be involved in providing information for benefit-risk decisions throughout the lifecycle of a medicine, including the early and late stages of development and the regulatory review. However, questions remain as to the appropriate methodologies for obtaining this information, whether the information should be at the disease or product level, the identity of the company personnel responsible for acquiring the information and regulators’ and decision makers’ views regarding the integrity of data generated by companies for patients. In addition, other issues for discussion include methods for ensuring the separation of patient and product advocacy, the potential for industry and agency collaboration in patient issues and whether patient advocates represent all patients with a specific disease.
This workshop explored these issues by providing a perspective from various stakeholders in the development and review of new medicines with a particular emphasis on potential methodologies and opportunities and barriers to including patients’ perspectives on benefits and risks at both the disease and product level.
Workshop objectives
- Identify key methodologies that are being used to capture patient’s needs in relation to benefits and risks by companies and regulators at both the disease and product level.
- Discuss the potential opportunities for utilisation of current and new approaches as well as the hurdles in both acquiring patient’s views and subsequently how these are being incorporated into the benefit-risk assessment of new medicines
- Develop proposals for appropriate patient “voice” pathways in clinical development, identifying which methodologies can be used to achieve scientifically acceptable approaches for including patients’ perspectives in the construction of the benefit-risk decision.